An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion

Huntington’s Disease (HD) is a progressive neurodegenerative disorder caused by CAG trinucleotide repeat expansions in exon 1 of the huntingtin (HTT) gene. The mutant HTT (mHTT) protein causes neuronal dysfunction, causing progressive motor, cognitive and behavioral abnormalities. Current treatments...

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Detalles Bibliográficos
Autores principales: Keller, Caroline Gubser, Shin, Youngah, Monteys, Alex Mas, Renaud, Nicole, Beibel, Martin, Teider, Natalia, Peters, Thomas, Faller, Thomas, St-Cyr, Sophie, Knehr, Judith, Roma, Guglielmo, Reyes, Alejandro, Hild, Marc, Lukashev, Dmitriy, Theil, Diethilde, Dales, Natalie, Cha, Jang-Ho, Borowsky, Beth, Dolmetsch, Ricardo, Davidson, Beverly L., Sivasankaran, Rajeev
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8894458/
https://www.ncbi.nlm.nih.gov/pubmed/35241644
http://dx.doi.org/10.1038/s41467-022-28653-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8894458/
https://www.ncbi.nlm.nih.gov/pubmed/35241644
http://dx.doi.org/10.1038/s41467-022-28653-6

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